AGENDA 

DAY 1: TARGETS 

November 17, 2022 - 11:00 AM – 4:00 PM EST 

11:00-11:15 AM EST Welcome and Workshop Overview 

11:00 AM: Workshop Welcome 

Joni Rutter, PhD, Acting Director, National Center for Advancing Translational Sciences, National Institutes of Health 

11:05 AM: Workshop Overview 

Karlie Sharma, PhD, Program Director, National Center for Advancing Translational Sciences, National Institutes of Health 

11:15-12:00 PM EST Introduction to Day 1

Overcoming Long-Standing Therapeutic Challenges Using Emerging Technologies: The Development of Covalent Inhibitors of KRAS (G12C)

Brian Lanman, PhD, Director of Research, Amgen 

12:00-1:15 PM EST Session 1: RNA 

12:00 PM: Introduction to Session 

12:05 – 12:45 PM: Ten Minute Talks

Design of Small Molecules Targeting RNA Structure

Chair: Matthew Disney, PhD, Chair, Department of Chemistry, University of Florida Scripps Biomedical Research 

RNAs as Drug Targets: The Uniqueness of the Ribosome

Adegboyega Oyelere, PhD, Associate Professor, Georgia Institute of Technology 

piRNAs as Regulators of Trypanosoma Cruzi Pathogenesis

Pius Nde, PhD, Assistant Professor, Meharry Medical College 

Targeting Pre-mRNA Splicing to Discover and Develop Novel Small Molecule Therapeutics

Anu Bhattacharyya, PhD, Executive Director Biology, PTC Therapeutics 

12:45 PM: Discussion 

1:15 – 1:30 PM EST Break 

1:30 – 2:45 PM EST Session 2: Protein Stability 

1:30 PM: Introduction to Session 

1:35 – 2:15 PM: Ten Minute Talks

How Prions Escape Drug Therapy

Chair: Surachai Supattapone, MD, PhD, Professor, Geisel School of Medicine, Dartmouth College 

Molecular Structures Reveal Synergistic Rescue of Δ508 CFTR by Trikafta Modulators

Jue Chen, PhD, Professor, The Rockefeller University

Making the Undruggable Druggable: Molecular Simulation as Computational Microscopy for Target Detection in Disordered and Liquid-Like Assemblies

Gül Zerze, PhD, Assistant Professor, University of Houston 

The Quest for Disease Modifying Treatments for Neurodegenerative Disorders

M. Maral Mouradian, MD, Director, RWJMS Institute for Neurological Therapeutics, Rutgers University 

2:15 PM: Discussion 

2:45 – 4:00 PM EST Session 3: Other Novel Targets 

2:45 PM: Introduction to Session 

2:50 – 3:30 PM: Ten Minute Talks 

Pivotal Role of Cholesterol Rich Lipid Rafts in Organizing the Dynamics of Membrane Signaling Proteins

Chair: Tony Yaksh, PhD, Professor, Department of Anesthesiology, University of California San Diego 

Targeting Golgi Defects for Treatment of Alzheimer’s Disease

Yanzhuang Wang, PhD, Professor, University of Michigan

Drug Screening for Keratinopathies and Porphyrias

Bishr Omary, MD, PhD, Professor of Medicine, Rutgers University 

Patient-Specific Stem Cell Models for Intractable Mitochondrial Disorders

Shilpa iYer, PhD, Associate Professor, University of Arkansas 

3:30 PM: Discussion 

4:00 PM EST Day 1 Workshop Wrap-Up and Adjourn 

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DAY 2: MODALITIES 

November 18, 2022 - 11:00 AM – 4:00 PM EST 

11:00 – 11:15 AM EST Welcome to Day 2 

Karlie Sharma, PhD, Program Director, National Center for Advancing Translational Sciences, National Institutes of Health 

11:15 – 12:00 PM EST Introduction to Day 2

Site-Directed Discovery and Covalent Leads for Difficult Targets

Michelle Arkin, PhD, Department Chair and Professor, University of California San Francisco  

12:00 – 1:15 PM EST Session 4: Repurposing Modalities 

12:00 PM: Introduction to Session 

12:05 – 12:45 PM: Ten Minute Talks 

The New World of Induced Proximity

Chair: Derek Lowe, PhD, Director in Chemical Biology and Therapeutics, Novartis 

The Next Frontiers for PROTAC® Protein Degraders

Ian Taylor, PhD, Chief Scientific Officer, Arvinas 

Expansion of the Druggable Genome Through Molecular Glue

Mary Matyskiela, PhD, Vice President, Molecular Science at Neomorph  

Deubiquitinase-Targeting Chimeras for Targeted Protein Stabilization

Lydia Boike, PhD, Scientist, Vicinitas Therapeutics  

12:45 PM: Discussion 

1:15 – 1:30 PM EST Break 

1:30 – 2:45 PM EST Session 5: Novel Modalities 

1:30 PM: Introduction to Session   

1:35 – 12:15 PM: Ten Minute Talks

Novel Modalities: Hope and Hurdles

Chair: Melissa Junttila, PhD, Vice President, ORIC Pharmaceuticals 

Rewriting Cellular Post-Translational Modifications for Challenging Targets

Christina Woo, PhD, Associate Professor, Harvard University 

Cracking the Code of Macrocycle Drug Discovery

Christian Cunningham, PhD, Director and Principal Scientist, Genentech 

Cystine-Dense-Peptides (CDP) as Therapeutic and Diagnostic Modalities

James Olson, MD, PhD, Professor, Fred Hutchinson Cancer Research Center

2:15 PM: Discussion 

2:45 – 4:00 PM EST Session 6: Novel Drug Delivery 

2:45 PM: Introduction to Session 

2:50 – 3:30 PM: Ten Minute Talks 

Novel Therapeutics and Delivery Mechanisms

Chair: Xandra Breakefield, PhD, Professor, Massachusetts General Hospital, Harvard Medical School 

Novel Bioengineered Microbiome-Based Therapeutic Delivery for Neurodegenerative Diseases

Anu Kanthasamy, PhD, Director, Center for Brain Science and Neurodegenerative Disorders, University of Georgia 

Non-Viral RNA and Genome Editing Delivery

Daniel Anderson, PhD, Professor, Koch Institute at MIT 

Amplifying Targetable Surface Areas Through New Delivery Approaches

Juliane Nguyen, PhD, Associate Professor, University of North Carolina 

3:30 PM: Discussion 

4:00 PM EST Workshop Wrap-Up and Adjourn